Adjuvant high-dose chemotherapy (HDCT) with hematopoietic stem cell transplant (HSCT) may be beneficial for patients with breast cancer and more than 9 involved axially lymph nodes (ALN), according to a study presented at the 2018 ESMO Congress in Munich, Germany.1

Though conventional chemotherapy is preferred over HDCT in most patients with high-risk early breast cancer due to toxicity, there may be a benefit for very high-risk patients. The aim of this study was to evaluate long-term outcomes of women who received HDCT and HSCT in a phase 3 trial.

The phase 3 trial was conducted between 1993 to 1999 and randomly assigned 885 women younger than 56 with early breast cancer and at least 4 involved ALN to receive conventional chemotherapy with fluorouracil, epirubicin, and cyclophosphamide or HDCT followed by autologous HSCT. The HDCT regimen was similar to the conventional regimen but replaced the last cycle with high-dose cyclophosphamide, thiotepa, and carboplatin.

In the overall cohort, there was no difference in relapse or death between the arms, with rates of 61% and 58% in the conventional chemotherapy and HDCT arms, respectively after a median follow-up of 20 years. Outcomes were also similar among patients with ER-positive or HER2-positive disease.

Among patients with more than 9 involved ALN, however, HDCT significantly improved outcomes. The relapse-free survival rate (RFS) was 39% in patients who received HDCT compared with 27% after conventional chemotherapy. HDCT also significantly prolonged overall survival (OS), with a 20-year survival rate of 44% compared with 30% with conventional chemotherapy.